by Huihao Tang, Chuanjun Shu, Haidi Chen, Xiaojing Zhang, Zhuqing Zang, Cheng Deng G protein–coupled receptors (GPCRs) play an important role in physiology and disease and represent the most productive...

[ad_1] Mundipharma EDO and Imbrium Therapeutics announced that the FDA granted orphan drug designation to etoposide toniribate for the treatment of cholangiocarcinoma, according to a press release.“We are pleased that...

[ad_1] Calliditas Therapeutics announced that the FDA has granted the company two orphan drug designations, one for an autoimmune hepatitis therapy and one for a primary biliary cholangitis therapy, according...

[ad_1] This article was originally published at https://www.drugpatentwatch.com/blog/mark-cuban-unexpected-leader-in-orphan-drug-development-reform/ Businessman and investor Mark Cuban is probably best known as the owner of the Dallas Mavericks and as an investor on the...

[ad_1] Albireo Pharma received FDA orphan designation for its lead product candidate A4250, an ileal bile acid transporter inhibitor for the treatment of biliary atresia, according to a press release.A4250...

[ad_1] This article was originally published at https://www.drugpatentwatch.com/blog/key-findings-from-gao-report-on-orphan-drug-approval-modernization/ In November 2018, the Government Accountability Office (GAO) released a report titled “FDA Could Improve Designation Review Consistency; Rare Disease Drug Development...

[ad_1] The European Commission has granted an orphan drug designation to Therachon’s apraglutide for the treatment of short bowel syndrome, according to a company press release.Apraglutide (FE 203799) – a...

[ad_1] Albireo Pharma received European Commission orphan designation for its lead product candidate A4250, an ileal bile acid transporter inhibitor for the treatment of biliary atresia, according to a press...

[ad_1] The FDA granted orphan drug designation to M7824 for the treatment of biliary tract cancer.This is the first regulatory designation for M7824 (Merck/EMD Serono), an investigational bifunctional immunotherapy designed...

[ad_1] Pliant Therapeutics received an FDA orphan drug designation for PLN-74809, an anti-fibrotic compound for treating primary sclerosing cholangitis, according to a press release.“Our excitement for lead product candidate PLN-74809...

[ad_1] This article was originally published at https://www.drugpatentwatch.com/blog/orphan-drug-patents-concerns-raised-by-payers-and-policy-makers/ There are more than 7,000 diseases that are considered “rare,” that is, affecting fewer than 200,000 Americans. Yet around one in ten...